Rare Daily Staff

Orphan drug accelerator Cydan said it has launched Tiburio Therapeutics to focus on developing treatments for rare neuroendocrine tumors and rare endocrine diseases and that the new company is backed with $31 million in funding.

Tiburio also named Abraham Ceesay as its CEO. Ceesay brings more than 17 years of  biopharmaceutical industry experience. Most recently, he served as chief operating officer of scPharmaceuticals.

Tiburio is developing two compounds licensed from Ipsen for the treatment of non-functioning pituitary adenoma (NFPA). NFPA is a non-metastatic pituitary tumor that arise from gonadotroph cells in the pituitary. Patients often present with symptoms related to the size of the NFPA tumor, including headaches, vision problems, changes in milk production, weight change, menstrual changes, fatigue, sleep disruption, and depression or cognitive/behavioral changes.

Current treatment is limited to transsphenoidal surgery, which is performed through the nose and sinus passage, and/or radiation to halt the growth of these tumors. However, these treatment options come with high-risk. In addition to these procedures, patients often require a complex set of medications to manage the endocrine disruption and other symptoms caused by the tumors and side effects of surgery and radiation.

In the U.S. there are approximately 5,000 new cases of NFPA that require TSS each year and 40-50 percent of these tumors will regrow within the first five years after surgery. Additionally, there are approximately 50,000 patients living with NFPA who may benefit from therapy to control tumor growth.

Tiburio’s lead experimental drug is TBR-760, a dopamine-somatostatin chimeric molecule. It inhibits NFPA cell proliferation and has the potential to shrink or halt tumor growth. Tiburio expects to move TBR-760 into a phase 2 clinical trial in patients with NFPA in the second half of 2019.

“Patients suffering from rare neuroendocrine tumors and endocrine diseases represent a significantly underserved patient population due to the lack of effective treatment options,” said Tiburio’s Ceesay. “TBR-760 and TBR-065 represent potential breakthroughs in the treatment of debilitating neuroendocrine diseases and we, at Tiburio, are intently focused on advancing these compounds for the benefit of patients.”

Tiburio is the third orphan drug company launched by Cydan, an orphan drug accelerator dedicated to creating therapies to improve the lives of patients living with orphan diseases.

January 3, 2018
Photo: Abraham Ceesay, CEO of Tiburio